CROSS-SECTIONAL OBSERVATIONAL STUDY OF LOW BONE MASS IN THALASSEMIA

地中海贫血低骨量的横断面观察研究

• 批准号: 7380725
• 负责人: INGRID A HOLM
• 金额: $ 0.07万
• 依托单位: BOSTON CHILDREN'S HOSPITAL
• 依托单位国家: 美国
• 财政年份: 2006
• 资助国家: 美国
• 起止时间: 2006-04-01 至 2007-03-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7380725
• 关键词: CROSS; SECTIONAL; OBSERVATIONAL; STUDY; LOW

This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. The purpose of this research is to estimate the prevalence of low bone mass among patients with thalassemia participating in the Thalassemia Clinical Research Network (TRCN), and to evaluate the interaction of potential modifying factors in bone metabolism in hopes of identifying preventative strategies and effective treatment regimens. Despite the severity of the low bone mass in thalassemia, there is little understanding of its pathogenesis which mandates comprehensive investigation. Reports of low bone mass in thalassemia have primarily been studied in adults with transfusion dependent B-thalassemia major, although more recent evidence suggests low bone mass affects children as well as adolescents. Bone mass in non-transfused thalassemia intermedia phenotypes such as B-E thalassemia and hemoglobin H disease (HbH) has not been well studied and may provide insight into the spectrum of low bone mass in thalassemia. We propose to assess measures of bone mass and bone turnover markers with potential patholphysiologic correlates. We suspect certain variables such as the thalassemia genotype, the degree of anemia, and ineffective erythropoiesis may predispose to low bone mass. Inadequate transfusion, iron chelation therapies, and endocrinopathies associated with iron overload may also act as potential contributors to low bone mass. The cross sectioal study will estimate the prevalence of low bone mass in thalassemia patients who are treated by investigators in the TCRN as well as provide a foundation for understanding its pathogenesis. It will also complement the future TCRN randomized trial of bisphosphonate therapy in thalassemia.

这个子项目是利用由NIH/NCRR资助的中心拨款提供的资源的许多研究子项目之一。子项目和调查员(PI)可能从另一个NIH来源获得了主要资金，因此可能会出现在其他CRISE条目中。列出的机构是针对中心的，而不一定是针对调查员的机构。本研究的目的是评估参与地中海贫血临床研究网络(TRCN)的地中海贫血患者中低骨量的患病率，并评估潜在的骨代谢调节因素之间的相互作用，以期确定预防策略和有效的治疗方案。尽管地中海贫血存在严重的低骨量，但其发病机制尚不清楚，需要进行全面的研究。有关地中海贫血骨量低的报道主要是在患有输血依赖型B-地中海贫血的成年人中进行的，尽管最近的证据表明，低骨量影响儿童和青少年。未输血的地中海贫血中间表型，如B-E地中海贫血和血红蛋白H病(HBH)的骨量尚未得到很好的研究，这可能有助于深入了解地中海贫血的低骨量谱系。我们建议评估具有潜在病理生理相关性的骨量和骨转换标记物。我们怀疑某些变量，如地中海贫血基因、贫血程度和无效的红细胞生成，可能是低骨量的易感因素。输血不足、铁离子螯合疗法以及与铁超载相关的内分泌疾病也可能是导致骨量减少的潜在因素。这项横断面研究将评估接受TCRN研究人员治疗的地中海贫血患者中低骨量的患病率，并为了解其发病机制提供基础。它还将补充未来的TCRN双膦酸盐治疗地中海贫血的随机试验。